Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
- Resource Type
- Article
- Authors
- Muñoz, Sergio; Bertolin, Joan; Jimenez, Veronica; Jaén, Maria Luisa; Garcia, Miquel; Pujol, Anna; Vilà, Laia; Sacristan, Victor; Barbon, Elena; Ronzitti, Giuseppe; El Andari, Jihad; Tulalamba, Warut; Pham, Quang Hong; Ruberte, Jesus; VandenDriessche, Thierry; Chuah, Marinee K.; Grimm, Dirk; Mingozzi, Federico; Bosch, Fatima
- Source
- In Molecular Metabolism March 2024 81
- Subject
- Language
- ISSN
- 2212-8778